siRNA and Gene Silencing Quiz
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Questions and Answers

What are the two main processes involved in the production of proteins from DNA?

Transcription and translation.

What does siRNA stand for?

Small interfering RNA.

How does siRNA prevent protein production?

siRNA targets and assists in the destruction of mRNA, preventing the translation of proteins.

What is the role of DICER in the antiviral defense system?

<p>DICER cuts viral RNA into smaller pieces, generating siRNA.</p> Signup and view all the answers

What is RISC, and what is its function in the siRNA pathway?

<p>RISC is the RNA-induced silencing complex, a multi-protein complex that binds to siRNA and targets it to complementary mRNA sequences.</p> Signup and view all the answers

What is the final step in the siRNA-mediated antiviral defense system?

<p>The siRNA/RISC complex cuts the mRNA, preventing protein production and leading to gene silencing.</p> Signup and view all the answers

What year did Andrew Fire and Craig Mello receive the Nobel Prize?

<ol start="2006"> <li></li> </ol> Signup and view all the answers

What is the key difference between siRNA and mRNA?

<p>siRNA is double-stranded, while mRNA is single-stranded.</p> Signup and view all the answers

What is the primary function of siRNA in gene silencing?

<p>siRNA works by targeting and degrading specific mRNA molecules, preventing the synthesis of the corresponding protein.</p> Signup and view all the answers

Describe the role of the RISC complex in siRNA-mediated gene silencing.

<p>The RISC complex binds to one strand of siRNA and guides it to the complementary mRNA target, ultimately facilitating its degradation.</p> Signup and view all the answers

What are two main methods for delivering siRNA into cells?

<p>siRNA can be delivered into cells using artificial approaches like liposomes or by viral-based vectors.</p> Signup and view all the answers

What is the primary benefit of utilizing siRNA technology in gene silencing?

<p>siRNA technology offers the potential to specifically target and downregulate the expression of any protein that we wish.</p> Signup and view all the answers

Identify three key challenges that currently limit the widespread use of siRNA therapeutics.

<p>Challenges include effective delivery of siRNA to the target site, ensuring successful entry into the cells, and maintaining the stability of siRNA in the body.</p> Signup and view all the answers

Mention two specific therapeutic areas where siRNA technology has shown promise.

<p>siRNA has shown potential in treating age-related macular degeneration and cancer.</p> Signup and view all the answers

What was the key event that marked the FDA approval of the first siRNA-based therapy?

<p>In 2018, Alnylam received FDA approval for its siRNA drug targeting mutations in the transthyretin gene, leading to a breakthrough in this field.</p> Signup and view all the answers

Discuss factors that might explain the initial enthusiasm for siRNA technology, followed by a period of slowed progress.

<p>Initial enthusiasm for siRNA was driven by its potential for targeted gene silencing, but challenges in delivery, efficacy, and off-target effects led to a period of slowed progress. Only recently have advancements in these areas allowed for the development of successful siRNA-based therapies.</p> Signup and view all the answers

Flashcards

siRNA

Small interfering RNA that targets mRNA to inhibit protein production.

Transcription

The process of creating mRNA from DNA in cells.

Translation

The process where mRNA is used to synthesize proteins.

Post-translational modification

Changes made to proteins after they are synthesized.

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DICER

An enzyme that cuts double-stranded RNA into smaller RNA fragments.

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RISC complex

RNA-induced silencing complex that interacts with siRNA.

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Gene silencing

The process of preventing gene expression, often using siRNA.

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Andrew Fire and Craig Mello

Scientists who discovered siRNA and won the Nobel Prize in 2006.

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RISC

RNA-induced silencing complex that uses siRNA to target mRNA.

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Gene Targeting

The process of specifically identifying and modifying a gene.

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Synthesis of siRNA

Creating siRNA that matches the target mRNA sequence.

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Therapeutic Areas for siRNA

Conditions where siRNA can be used, such as cancers and asthma.

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Delivery Challenges

Issues related to getting siRNA to the right cells effectively.

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Study Notes

Small Interfering RNA (siRNA)

  • siRNA is a method to prevent protein production by targeting and destroying messenger RNA (mRNA).
  • siRNA molecules are short RNA fragments that inhibit protein translation.
  • siRNA works by targeting mRNA using a RISC complex, which creates a cut that prevents the translation of the protein.
  • siRNA's mechanism is similar to the body's antiviral defense system, using DICER to break down viral RNA into smaller fragments.

siRNA's Mechanism of Action

  • siRNA sequence is designed to match the mRNA encoding the protein being targeted.
  • siRNA is typically double-stranded RNA, around 20 base pairs long, and commercially synthesized.
  • The siRNA is introduced into cells using artificial or viral-based methods.
  • The RISC complex (containing Ago2 protein) binds one strand of siRNA.
  • The siRNA/RISC complex targets and cuts the specific mRNA.
  • The cut mRNA is no longer able to create the protein, leading to gene silencing.

siRNA Applications and Challenges

  • siRNA targets various tissues, including eyes, lungs, liver, skin, kidney, and tumours.

  • siRNA treatment areas include age-related macular degeneration, asthma, metabolic diseases, dermatological conditions, renal failure, and cancer.

  • siRNA technology showed promise in the past. Merck acquired Sirna Therapeutics in 2006 but later sold it to Alnylam at a lower price.

  • Novartis sold its RNAi business to Arrowhead at a low cost.

  • In 2018, Alnylam's siRNA was approved by the FDA for treating a rare disease.

  • Main benefit: Ability to design specific siRNA for each protein due to our knowledge of the human genetic code enabling precise downregulation of drug targets.

  • Main challenges: siRNA delivery to target cells, cellular uptake, and siRNA stability.

  • Novel methods are being developed to overcome these delivery challenges.

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Description

Test your understanding of small interfering RNA (siRNA) and its role in gene silencing. This quiz covers key concepts, mechanisms, and challenges related to siRNA technology, as well as its therapeutic applications. Perfect for students and professionals interested in molecular biology and genetics.

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